（文章来源：21世纪经济报道） 昌平实验室科学家刘河生教授团队2月5日发布，团队发现了帕金森病的底层功能网络机制，并针对这一 网络进行直接干预，在临床研究中取得良好成效，此次发现属世界首次。这项突破性成果发表于5日上 线的国际学术期刊《自然》。 ...

中国团队领衔研究：靶向新发现的大脑网络或改善帕金森病治疗 中新网北京2月5日电 (记者 孙自法)国际知名学术期刊《自然》最新发表一篇神经科学论文称，一个研 究新发现的连接认知(思考)与行动及身体状态的大脑网络——"躯体-认知动作网络"(SCAN)，或在帕金 森病中起到重要作用并有望改善其疗法。 由中国科研团队领衔的这项国际合作研究发现，相较于靶向其他运动脑区，靶向"躯体-认知动作网 络"有望使部分帕金森病现有疗法的有效性翻倍，这提示该网络是解释帕金森病广泛症状的一个潜在候 选机制，或为帕金森病一个关键的治疗靶点。 编辑：张嘉怡 广告等商务合作，请点击这里 本文为转载内容，授权事宜请联系原著作权人 中新经纬版权所有，未经书面授权，任何单位及个人不得转载、摘编或以其它方式使用。 关注中新经纬微信公众号(微信搜索"中新经纬"或"jwview")，看更多精彩财经资讯。 据介绍，帕金森病不仅会影响行动和睡眠这类身体机能，也会影响动力和思维能力。尽管颤抖、僵硬和 步态障碍是帕金森病的典型特征，但许多症状并未发现与特定身体部位的关联。 在本项研究中，北京大学刘河生教授、昌平实验室科学家任建勋联合美国华盛顿大学医学院Nico ...

来源：新华社 我国科学家世界首次发现帕金森病底层功能网络机制 昌平实验室科学家刘河生教授团队2月5日发布，团队发现了帕金森病的底层功能网络机制，并针对这一网络进行直接干预，在临床研究中取得 良好成效，此次发现属世界首次。这项突破性成果发表于5日上线的国际学术期刊《自然》。 据介绍，帕金森病是一种常见的神经退行性疾病，患者常表现出震颤、动作迟缓、肌肉僵硬等症状，其治疗面临诸多挑战。 据悉，这一研究由昌平实验室牵头，联合北京大学、清华大学、河南省人民医院等国内外10余家机构共同完成。 记者：熊琳、王修楠 帕金森病躯体认知环路示意图。（受访单位供图） "这一发现将更新我们对帕金森病的了解，同时为开辟新的治疗方法提供了思路。"刘河生表示，环路的发现能够揭示更多的潜在治疗靶点，并 催生新的治疗方式。 根据这一机制，刘河生团队自主研发出新一代精准脑环路刺激系统，实现了帕金森病治疗靶点的精准定位与无创治疗，设备已获批国家医疗器 械注册证。临床结果显示，在治疗2周后，躯体认知靶点组的治疗有效率达55.5%，而刺激传统的大脑运动区有效率为22.2%。 昌平实验室主任、中国科学院院士谢晓亮表示，昌平实验室作为国家战略科技力量，旨 ...

2026.01. 26 帕金森病是仅次于阿尔茨海默病的第二常见的神经退行性疾病，严重影响患者日常生活，致残率和死亡率较 高。我国每年死于帕金森病的人数超过7万人。 现有的传统药物和手术治疗都只针对帕金森病的症状进行治疗，不能延缓疾病进展，因而进一步研究帕金森 病的致病原理并开展针对性治疗，成为全球相关领域科学家竞相探索的战略高地。 本文字数：1044，阅读时长大约2分钟 郁金泰团队利用人工智能的蛋白结构预测和虚拟筛选技术，从7000余种药物中成功找到了一种针对帕金森 病的候选新药bemcentinib，并通过体外实验和动物实验证实小分子bemcentinib可有效抑制帕金森病的关 键靶点FAM171A2蛋白和病理性α-突触核蛋白结合，从而抑制多巴胺能神经元对该致病蛋白纤维的摄取。 作者 | 第一财经 钱童心 现有的传统药物和手术治疗都只针对帕金森病的症状进行治疗，不能延缓疾病进展，因而进一步研究帕金森 病的致病原理并开展针对性治疗，成为全球相关领域科学家竞相探索的战略高地。 1月26日，两院院士揭晓2025十大科技进展。其中，复旦大学研究院副院长、复旦大学附属华山医院神经 内科郁金泰团队在全球首次发现的帕金森 ...

Core Insights - The 2025 China and World Top Ten Scientific Advances were announced, highlighting significant achievements in various fields of science and technology [2][13]. Group 1: China's Top Ten Scientific Advances - The "artificial sun" EAST achieved a world record of 1 billion degrees Celsius for 1066 seconds, marking a major leap in fusion energy research [14]. - DeepSeek launched a new AI model R1, which matches the performance of OpenAI's models while significantly reducing training costs and being fully open-source [15]. - A thorium-based molten salt reactor was built, achieving thorium-uranium fuel conversion and demonstrating the feasibility of using thorium resources in nuclear energy [15]. - A high-precision AI diagnostic tool for predicting liver cancer recurrence was developed, achieving an accuracy rate of 82.2% and featured on the cover of Nature [16]. - The "North Brain No. 1" wireless brain-computer interface was successfully implanted, enabling patients with speech impairments to communicate effectively [17]. - A novel target for Parkinson's disease was discovered, providing new hope for treatment and drug development [18]. - The "Zu Chongzhi No. 3" superconducting quantum computer was built, outperforming the fastest supercomputers by a factor of 10 trillion [19]. - New hydrogen production technology was developed, eliminating CO2 emissions and achieving high hydrogen yield [20]. - Significant breakthroughs in black soil protection technology were reported, supporting sustainable agricultural practices [21]. - A new ultra-wideband optoelectronic integrated system for 6G communication was developed, enabling high-speed wireless communication across a wide frequency range [22]. Group 2: World Top Ten Scientific Advances - Brain-computer interfaces allow patients to express emotions through speech and song, translating thoughts into language in real-time [23]. - An integrated chip system combining electronics, photonics, and quantum technology was created [23]. - The largest black hole merger event was detected, challenging existing black hole formation models [23]. - The highest energy neutrino ever detected was found, being 20 times more energetic than previous detections [23]. - The first visible "time crystal" was created [23]. - Transgenic pig organ transplants set a new record for survival time [23]. - Ground-based telescopes detected signals from 13 billion years ago [23]. - The largest cosmic map to date was produced [23]. - The most detailed brain map was created, describing numerous neurons and their activities [23]. - "Deep Thinking" announced AI test scores equivalent to gold medal levels in international mathematics competitions [23].

郁金泰团队利用人工智能的蛋白结构预测和虚拟筛选技术，从7000余种药物中成功找到了一种针对帕金 森病的候选新药bemcentinib，并通过体外实验和动物实验证实小分子bemcentinib可有效抑制帕金森病的 关键靶点FAM171A2蛋白和病理性α-突触核蛋白结合，从而抑制多巴胺能神经元对该致病蛋白纤维的摄 取。 现有的传统药物和手术治疗都只针对帕金森病的症状进行治疗，不能延缓疾病进展，因而进一步研究帕 金森病的致病原理并开展针对性治疗，成为全球相关领域科学家竞相探索的战略高地。 1月26日，两院院士揭晓2025十大科技进展。其中，复旦大学研究院副院长、复旦大学附属华山医院神 经内科郁金泰团队在全球首次发现的帕金森病全新治疗靶点FAM171A2及基于该靶点的候选药物入选 2025中国十大科技进展。 帕金森病是仅次于阿尔茨海默病的第二常见的神经退行性疾病，严重影响患者日常生活，致残率和死亡 率较高。我国每年死于帕金森病的人数超过7万人。 现有的传统药物和手术治疗都只针对帕金森病的症状进行治疗，不能延缓疾病进展，因而进一步研究帕 金森病的致病原理并开展针对性治疗，成为全球相关领域科学家竞相探索的战略高地。 郁金 ...

Summary of Gain Therapeutics Conference Call Company Overview - **Company**: Gain Therapeutics (NasdaqGM:GANX) - **Focus**: Development of GT02287 for Parkinson's disease treatment Key Industry Insights - **Parkinson's Disease Prevalence**: Second most prevalent neurodegenerative disease in the U.S. with approximately 1 million patients, 15% of whom carry a GBA1 mutation [8][2] - **GCase Activity**: The enzyme glucocerebrosidase (GCase) is crucial in the pathophysiology of Parkinson's, with mutations leading to reduced enzyme activity and associated disease progression [12][18] Core Findings from the Conference Call - **Phase 1b Study Results**: - Patients showed an average improvement of 2.2 points in MDS-UPDRS scores after 90 days of treatment with GT02287 [2][10] - The improvement aligns with expectations based on the drug's mechanism of action [3] - A clinically meaningful improvement is considered to be approximately six points over a year compared to placebo [3] - **Biomarker Evidence**: - GT02287 modulates GCase activity, leading to a reduction in glucosylsphingosine, a toxic substrate implicated in Parkinson's [4][5] - Stabilization of MDS-UPDRS scores observed without changes in dopamine administration suggests a potential disease-modifying effect [5][42] - **Study Design**: - The phase 1b study included a 90-day treatment followed by an optional open-label extension [8][26] - 21 patients enrolled, with 19 completing the first part of the study [26] - **Future Expectations**: - Continued analysis of phase 1b study extension and safety [42] - Final results expected in the second half of 2026, with plans to initiate a phase II study [43][44] Important Considerations - **Glucosylsphingosine as a Biomarker**: - Emerging evidence suggests glucosylsphingosine is a better biomarker for treatment response than glucosylceramide [35][36] - Measurement in cerebrospinal fluid (CSF) is critical for assessing central nervous system effects [36] - **Regulatory Pathway**: - Discussions with the FDA regarding the use of glucosylsphingosine as a surrogate biomarker are ongoing, but traditional UPDRS-based studies may still be required for approval [55][56] - **Patient Population**: - Future studies may focus on patients with specific glucosylsphingosine levels or GBA1 mutations, as they may respond better to treatment [46][49] Additional Insights - **Mechanism of Action**: - The drug aims to increase GCase activity, potentially leading to reduced glucosylsphingosine levels, which could have long-term benefits for Parkinson's patients [34][38] - **Clinical Implications**: - The reduction of glucosylsphingosine in CSF correlates with improvements in UPDRS scores, indicating a possible link between biomarker levels and clinical outcomes [41][45] This summary encapsulates the key points discussed during the conference call, highlighting the company's focus on GT02287, the significance of biomarker evidence, and the implications for future clinical development and regulatory interactions.

Core Insights - The launch of Entacapone/Carbidopa/Levodopa tablets (brand name: Shenkang) by Qilu Pharmaceutical Group marks a significant advancement in the treatment of Parkinson's disease, particularly for patients experiencing motor fluctuations not controlled by standard therapies [1][2] Group 1: Product Overview - Shenkang is indicated for adult patients with Parkinson's disease who experience motor fluctuations despite treatment with Levodopa and DDC inhibitors [1] - The drug combines three active ingredients—Levodopa, Carbidopa, and Entacapone—into a single formulation, simplifying the treatment regimen and potentially improving patient adherence [1] - The Chinese Parkinson's Disease Treatment Guidelines recommend this combination therapy as the first-line treatment for patients with motor fluctuations [1] Group 2: Market Context - Parkinson's disease is the second most common neurodegenerative disorder in China, with a prevalence rate of approximately 1.7% among individuals aged 65 and older [1] - Qilu Pharmaceutical is a major player in the Chinese pharmaceutical industry, with a diverse product portfolio covering various therapeutic areas including oncology, infectious diseases, and central nervous system disorders [2] Group 3: Distribution and Management - Alibaba Health serves as the launch platform for Shenkang, providing a pathway for rapid access to target patients and enhancing disease management through professional medical education and pharmacy services [2] - The company emphasizes a comprehensive management approach for Parkinson's disease, utilizing digital tools for online follow-ups, medication reminders, and symptom tracking to improve treatment continuity and patient quality of life [2]

Core Viewpoint - The approval of the controlled-release formulation of Carbidopa-Levodopa by the National Medical Products Administration enhances the company's product pipeline in the Parkinson's disease treatment sector, particularly with the introduction of a first generic version in the domestic market [1] Group 1: Product Approval - The company received two drug registration certificates for Carbidopa-Levodopa controlled-release tablets [1] - The specifications for the approved products include Carbidopa 50mg and Levodopa 200mg, and Carbidopa 25mg and Levodopa 100mg [1] - The latter specification is the first generic version in China, as the original product has not been registered for sale domestically [1] Group 2: Market Impact - The approval enriches the company's offerings in the Parkinson's disease treatment market, providing a significant opportunity in a niche segment [1] - The controlled-release formulation is recognized as the "gold standard" for treating Parkinson's disease according to the "Chinese Parkinson's Disease Treatment Guidelines (2020 Edition)" [1] - The availability of the smaller dosage form improves medication convenience for patients with mild symptoms [1]

Core Viewpoint - The company has received approval from the National Medical Products Administration for two drug registration certificates for Carbidopa-Levodopa Extended-Release Tablets, enhancing its product pipeline in the Parkinson's disease treatment sector [1] Group 1: Product Approval - The Carbidopa-Levodopa Extended-Release Tablets (specification: Carbidopa 50mg, Levodopa 200mg) is the third approved product in the domestic market [1] - The Carbidopa-Levodopa Extended-Release Tablets (specification: Carbidopa 25mg, Levodopa 100mg) is the first generic version in the domestic market, with no original product registered for sale [1] Group 2: Market Opportunities - The approval of these products expands the company's opportunities in the Parkinson's disease treatment market [1] - The new products may lead to increased business prospects in this niche market [1] Group 3: Sales Uncertainty - The production and sales of the drugs may be influenced by changes in the market environment and other comprehensive factors, leading to certain uncertainties in sales performance [1]